IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study)

• Status: Recruiting
• Trial end date: 2024-09-30
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to assess the safety and tolerability of two 5-day infusion cycles of IV gallium in adult patients with CF who are infected with NTM.

Funding Source - FDA OOPD

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Chris Goss
• Collaborator: Cystic Fibrosis Foundation
• Treatments: Gallium nitrate

Criteria

Inclusion Criteria:

1. Written informed consent obtained from subject or subject's legal representative

2. Be willing and able to adhere to the study visit schedule and other protocol requirements

3. Greater than or equal to 18 years of age at Visit 1

4. Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:

• Sweat chloride ≥ 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)

• Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene

• Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproteronol of less than -5 mV)

5. Documentation of NTM culture positive defined as follows:

• Two positive NTM culture results from sputum (or BAL) at least 28 days apart (these are the two qualifying positive cultures)

• Both qualifying positive culture results include M. avium complex, M. abscessus complex, or both M. avium and M. abscessus

• Both qualifying positive culture results include the same species or subspecies

• No cultures negative for NTM since the first of the two qualifying positive culture results

6. Current NTM species or subspecies has never been treated or previous treatment was associated with clearance of NTM and completed > 2 years prior to Day 1

7. Forced expiratory volume in 1 second (FEV1) ≥ 25 % of predicted value at Screening

8. Able to expectorate sputum

9. Clinically stable with no significant changes in health status within 7 days prior to Day 1

10. Enrolled in the CFF Cystic Fibrosis Foundation Patient Registry (CFFPR)

11. Willing to discontinue chronic azithromycin use for the duration of the study

Exclusion Criteria:

1. Any of the following abnormal lab values at screening:

• Hemoglobin <10g/dL

• Platelets <100,000/mm3

• White blood cells (WBC) < 4,500/mm3

• Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) ≥3 x upper limit of normal

• Serum creatinine > 2.0 mg/dl and ≥1.5 x upper limit of normal

• Ionized calcium ≤ lower limit of normal (only performed if total calcium is ≤ lower limit of normal)

2. History of solid organ or hematological transplantation

3. Use of bisphosphonates within 7 days prior to Day 1

4. Known sensitivity to gallium

5. Use of any investigational drug and/or participated in any interventional clinical trial within 28 days prior to Day 1

6. In the opinion of the Investigator, features of active NTM disease are present (e.g., clinical worsening is likely due to NTM disease despite definitive treatment of co-pathogens and/or acute exacerbations)

7. Undergoing treatment for NTM disease or anticipate beginning treatment within 3 months

8. Current diagnosis of osteoporosis

9. For people of childbearing potential:

• Positive pregnancy test at Visit 1 or

• Lactating or

• Unwilling to practice a medically acceptable form of contraception (acceptable forms of contraception: abstinence, hormonal birth control, intrauterine device, or barrier method plus a spermicidal agent), unless surgically sterilized or postmenopausal during the study

10. For people able to father a child: unwilling to use adequate contraception (as determined by the investigator) during the study

11. Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives

12. New initiation of chronic therapy (greater than 21 days) within 28 days prior to the Enrollment Visit